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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT04598477
Registration number
NCT04598477
Ethics application status
Date submitted
8/10/2020
Date registered
22/10/2020
Date last updated
24/04/2024
Titles & IDs
Public title
A Study to Assess the Long-term Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod PH20 SC in Adults With Pemphigus (Vulgaris or Foliaceus)
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Scientific title
An Open-Label, Multicenter, Follow-up Trial of ARGX-113-1904 to Evaluate the Safety, Tolerability, and Efficacy of Efgartigimod PH20 SC in Patients With Pemphigus
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Secondary ID [1]
0
0
ARGX-113-1905
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Universal Trial Number (UTN)
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Trial acronym
ADDRESS+
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Condition category
Condition code
Skin
0
0
0
0
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Dermatological conditions
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Skin
0
0
0
0
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Other skin conditions
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Inflammatory and Immune System
0
0
0
0
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Autoimmune diseases
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Other interventions - efgartigimod PH20 SC
Treatment: Drugs - prednisone
Experimental: efgartigimod PH20 SC - patients receiving efgartigimod PH20 SC on top of prednisone
Other interventions: efgartigimod PH20 SC
Subcutaneous injection of efgartigimod using rHuPH20 (PH20) as a permeation enhancer
Treatment: Drugs: prednisone
Oral prednisone tablets
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Intervention code [1]
0
0
Other interventions
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Intervention code [2]
0
0
Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Incidence of Treatment-Emergent Adverse Events (TEAE), Adverse Events of Special Interest (AESI), and Serious Adverse Events (SAE)
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Assessment method [1]
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Incidence of Treatment-Emergent Adverse Events (TEAE), Adverse Events of Special Interest (AESI), and Serious Adverse Events (SAE)
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Timepoint [1]
0
0
Up to 60 weeks
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Primary outcome [2]
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0
Severity of Treatment-Emergent Adverse Events (TEAE), Adverse Events of Special Interest (AESI), and Serious Adverse Events (SAE)
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Assessment method [2]
0
0
Severity of Treatment-Emergent Adverse Events (TEAE), Adverse Events of Special Interest (AESI), and Serious Adverse Events (SAE)
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Timepoint [2]
0
0
Up to 60 weeks
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Secondary outcome [1]
0
0
Proportion of Pemphigus Vulgaris (PV) participants who achieve complete clinical remission (CR) on minimal prednisone therapy
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Assessment method [1]
0
0
Proportion of participants with Pemphigus Vulgaris who achieve complete clinical remission on minimal prednisone therapy.
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Timepoint [1]
0
0
Up to 52 weeks treatment period
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Secondary outcome [2]
0
0
Proportion of Pemphigus Vulgaris (PV) and Pemphigus Foliaceus (PF) participants who achieve complete clinical remission (CR) on minimal prednisone dose therapy
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Assessment method [2]
0
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Proportion of participants with Pemphigus Vulgaris and Pemphigus Foliaceus who achieve complete clinical remission on minimal prednisone therapy.
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Timepoint [2]
0
0
Up to 52 weeks treatment period
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Secondary outcome [3]
0
0
Time to Disease Control (DC)
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Assessment method [3]
0
0
Time to Disease Control
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Timepoint [3]
0
0
Up to 60 weeks
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Secondary outcome [4]
0
0
Time to complete clinical remission (CR)
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Assessment method [4]
0
0
Time to complete clinical remission
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Timepoint [4]
0
0
Up to 60 weeks
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Secondary outcome [5]
0
0
Time to complete clinical remission (CR) on minimal prednisone therapy
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Assessment method [5]
0
0
Time to complete clinical remission on minimal prednisone therapy
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Timepoint [5]
0
0
Up to 60 weeks
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Secondary outcome [6]
0
0
Time to complete clinical remission (CR) off prednisone therapy
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Assessment method [6]
0
0
Time to complete clinical remission off prednisone therapy
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Timepoint [6]
0
0
Up to 60 weeks
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Secondary outcome [7]
0
0
Time to flare
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Assessment method [7]
0
0
Time to flare
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Timepoint [7]
0
0
Up to 60 weeks
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Secondary outcome [8]
0
0
Rate of treatment failure
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Assessment method [8]
0
0
Rate of treatment failure
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Timepoint [8]
0
0
Up to 60 weeks
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Secondary outcome [9]
0
0
Rate of flare
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Assessment method [9]
0
0
Rate of flare
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Timepoint [9]
0
0
Up to 60 weeks
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Secondary outcome [10]
0
0
Cumulative prednisone dose over the trial
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Assessment method [10]
0
0
Cumulative prednisone dose over the trial
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Timepoint [10]
0
0
Up to 52 weeks treatment period
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Secondary outcome [11]
0
0
Pemphigus Disease Area Index (PDAI) at each visit
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Assessment method [11]
0
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Pemphigus Disease Area Index at each visit
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Timepoint [11]
0
0
Up to 52 weeks treatment period
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Secondary outcome [12]
0
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EuroQol 5-Dimension 5-Level (EQ-5D-5L) score
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Assessment method [12]
0
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EuroQol 5-Dimension 5-Level score
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Timepoint [12]
0
0
Up to 52 weeks treatment period
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Secondary outcome [13]
0
0
Autoimmune Blister Quality of Life (ABQOL) score
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Assessment method [13]
0
0
Autoimmune Blister Quality of Life score
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Timepoint [13]
0
0
Up to 52 weeks treatment period
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Secondary outcome [14]
0
0
Efgartigimod serum concentrations
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Assessment method [14]
0
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Efgartigimod serum concentrations
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Timepoint [14]
0
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Up to 60 weeks
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Secondary outcome [15]
0
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Total Immunoglobulin G and subtype (IgG1, IgG2, IgG3, IgG4) serum levels
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Assessment method [15]
0
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Total Immunoglobulin G and subtype (IgG1, IgG2, IgG3, IgG4) serum levels
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Timepoint [15]
0
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Up to 60 weeks
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Secondary outcome [16]
0
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Anti-desmoglein (Dsg) -1 and -3 autoantibodies serum levels
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Assessment method [16]
0
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Anti-desmoglein (Dsg) -1 and -3 autoantibodies serum levels
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Timepoint [16]
0
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Up to 60 weeks
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Secondary outcome [17]
0
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Incidence of antidrug antibodies (ADA) against efgartigimod PH20 SC
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Assessment method [17]
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Incidence of antidrug antibodies (ADA) against efgartigimod PH20 SC
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Timepoint [17]
0
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Up to 60 weeks
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Secondary outcome [18]
0
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Prevalence of antidrug antibodies (ADA) against efgartigimod PH20 SC
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Assessment method [18]
0
0
Prevalence of antidrug antibodies (ADA) against efgartigimod PH20 SC
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Timepoint [18]
0
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Up to 60 weeks
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Secondary outcome [19]
0
0
Composite Glucocorticoid Toxicity Index (C-GTI) comprising the Aggregate Improvement Score (AIS) and the Cumulative Worsening Score (CWS)
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Assessment method [19]
0
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Composite Glucocorticoid Toxicity Index comprising the Aggregate Improvement Score and the Cumulative Worsening Score
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Timepoint [19]
0
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Up to 52 weeks treatment period
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Secondary outcome [20]
0
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Percentage of participants who performed self-administration
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Assessment method [20]
0
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Percentage of participants who performed self-administration
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Timepoint [20]
0
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Up to 52 weeks
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Secondary outcome [21]
0
0
Percentage of caregivers who administered the injection to the participant
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Assessment method [21]
0
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Percentage of caregivers who administered the injection to the participant
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Timepoint [21]
0
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Up to 52 weeks
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Secondary outcome [22]
0
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Number of visits needed for the participant or caregiver to be competent to start administering efgartigimod PH20 SC
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Assessment method [22]
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Number of visits needed for the participant or caregiver to be competent to start administering efgartigimod PH20 SC
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Timepoint [22]
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Up to 52 weeks
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Secondary outcome [23]
0
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Frequency of self- or caregiver-supported administration at home
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Assessment method [23]
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Frequency of self- or caregiver-supported administration at home
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Timepoint [23]
0
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Up to 52 weeks
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Eligibility
Key inclusion criteria
1. Ability to understand the requirements of the trial, to provide written informed
consent (including consent for the use and disclosure of research-related health
information), willingness and ability to comply with the trial protocol procedures
(including required trial visits).
2. The participant participated in trial ARGX-113-1904 and completed the study or has the
defined criteria for rollover.
3. Contraceptive use by men and women should be consistent with local regulations
regarding the methods for contraception for those participating in clinical trials
and:
1. Male participants:
Male participants must agree to use an acceptable method of contraception as
described in the protocol, from signing the ICF until the last dose of the study
drug.
2. Female participants
Women of childbearing potential (WOCBP) must:
- have a negative urine pregnancy test at baseline before the IMP can be administered,
- agree to use a highly effective or acceptable contraception method (as described in
the protocol), which should be maintained at minimum until after the last dose of IMP
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
1. Pregnant and lactating women and those intending to become pregnant during the trial.
2. Participants with clinical evidence of other significant serious disease or
participants who recently underwent or have planned a major surgery during the period
of the trial, or any other condition in the opinion of the investigator, that could
confound the results of the trial or put the participant at undue risk.
3. Known hypersensitivity to any of the components of the administered treatments.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
N/A
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Terminated
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
15/07/2021
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
25/03/2024
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Sample size
Target
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Accrual to date
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Final
183
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Recruitment in Australia
Recruitment state(s)
NSW,VIC
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Recruitment hospital [1]
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0
Investigator site 15 - AU0610006 - Sydney
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Recruitment hospital [2]
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Investigator site 11 - AU0610007 - Parkville
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Recruitment hospital [3]
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Investigator site 92 - AU0610013 - Melbourne
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Recruitment postcode(s) [1]
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2217 - Sydney
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Recruitment postcode(s) [2]
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3050 - Parkville
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Recruitment postcode(s) [3]
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3065 - Melbourne
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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Alabama
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Country [2]
0
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United States of America
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Arizona
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United States of America
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California
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United States of America
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Florida
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United States of America
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Minnesota
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United States of America
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Missouri
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New York
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United States of America
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North Carolina
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United States of America
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Ohio
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Country [10]
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United States of America
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Pennsylvania
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United States of America
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Texas
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United States of America
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Virginia
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Bulgaria
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Pleven
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Bulgaria
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Plovdiv
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Bulgaria
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Sofia
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Beijing
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Fuzhou
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Guangzhou
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Zhengzhou
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Serbia
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Belgrade
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Serbia
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Niš
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Serbia
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Novi Sad
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Barcelona
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Spain
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Granada
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Spain
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Spain
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Sevilla
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Turkey
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Gaziantep
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Turkey
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Istanbul
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Ukraine
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Dnipro
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Ukraine
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Ivano-Frankivs'k
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Country [93]
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Ukraine
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State/province [93]
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Kyiv
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Ukraine
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State/province [94]
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Lviv
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Ukraine
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Zaporizhzhia
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United Kingdom
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State/province [96]
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Birmingham
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United Kingdom
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State/province [97]
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Bristol
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Country [98]
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United Kingdom
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Southampton
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Funding & Sponsors
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Commercial sector/Industry
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Name
argenx
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Summary
Brief summary
This is a prospective, multicenter, open label extension (OLE) trial on the efficacy, safety, patient outcome measures, tolerability, immunogenicity, PK and PD of efgartigimod PH20 SC in adult PV or PF participants, who participated in antecedent trial ARGX-113-1904. This trial provides extension of efgartigimod PH20 SC treatment and retreatment options for participants who have been randomized to efgartigimod PH20 SC treatment arm in the trial ARGX-113-1904, and the first treatment of efgartigimod PH20 SC and retreatment options for participants who had been randomized to placebo arm in trial ARGX-113-1904. Trial ARGX-113-1905 evaluates ability to (further) taper prednisone therapy and achieve Clinical Remission (CR) off therapy (CRoff), the ability to achieve CR and CR on minimal therapy (CRmin) for participants who had not yet achieved CRmin, and the ability to treat flare; and assess patient outcome measures and the safety, PD, PK and immunogenicity of efgartigimod PH20 SC over the duration of trial. Study duration: Up to 60 weeks for participants who receive IMP administration up to 52 weeks and with a follow-up period of 8 weeks after the last IMP administration
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Trial website
https://clinicaltrials.gov/ct2/show/NCT04598477
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Contacts
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Contact person for scientific queries
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Results not provided in
https://clinicaltrials.gov/ct2/show/NCT04598477
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